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Gene Therapies: The Next Frontier in Patient-Focused Drug Development

Power of Ideas
Gene Therapies: The Next Frontier in Patient-Focused Drug Development

A biological revolution is underway. Gene therapies, which attempt to treat or cure diseases through the modification or manipulation of gene expression, are becoming a clinical reality, holding incredible promise for millions of patients. These therapies are evolving and proliferating at a rapid rate. In 2016, the Office of Tissues and Advanced Therapies at the US Food and Drug Administration (FDA), which covers gene therapies, received 163 investigational new drug applications; in 2020, that number jumped to 350 applications. As of October 2022, there were over 2,000 gene therapies in the development pipeline.

Incorporating the patient voice into the gene therapy development process is both an essential component to the success of these therapies and an ethical imperative. Gene therapies are incomparably personal and carry a significant level of uncertainty. They involve the incorporation of foreign DNA into a patient’s cells; riskier and more enduring gene therapies, such as genome editing, permanently alter a patient’s DNA.

Every patient who consents to receiving a gene therapy product puts a staggering amount of trust in product developers, regulators, and health-care providers. Including patients throughout the development process is more than an opportunity—it’s a necessity. And though it’s critical to assess risk tolerance, it isn’t enough to ask what patients will tolerate. We must ask patients what they want.

Stakeholders must be proactive to effectively incorporate patient goals and perspectives throughout the gene therapy development process.

The Cystic Fibrosis Foundation is dedicated to ensuring that people with cystic fibrosis (CF) have the opportunity to lead long, fulfilling lives, with the ultimate goal of curing CF. Since our founding, we have gained extensive experience with the value of patient-focused initiatives. We ensure that the foundation’s work is relevant and impactful by engaging people with CF as core advisors to our programs. Our Community Voice platform allows members of the CF community to partner with us, researchers, and other organizations to share their experiences and bring their insights and priorities to the forefront of CF research and care.

Through our listening and outreach, the Cystic Fibrosis Foundation has gathered invaluable information about how the CF community views gene therapies. Assessing awareness of gene therapies reveals educational opportunities and provides a roadmap to develop tools. Determining what patients consider to be major barriers to participation in clinical trials allows us to design more successful, accessible clinical trials. Learning what patients consider to be the most critical benefits of a gene therapy, or even what they would consider to be a cure, helps all those engaged in development to conceptualize what a CF gene therapy should look like in the first place.

This input—from people with the most at stake—shapes and improves our scientific work, including our research and development division and clinical trial network, and allows us to better meet the needs of people with CF. We believe that our experiences in CF may serve as an effective model for elevating and incorporating the patient voice in the context of other conditions and stages of the development process.

In his remarks at the 2022 National Organization for Rare Disorders Summit, FDA Commissioner Robert Califf, MD, emphasized the importance of building a patient-centered “ecosystem” when tackling scientific and medical challenges. We believe that it is the responsibility of developers, regulators, patient advocacy groups, and other stakeholders to put forth the effort to build this ecosystem and ensure that gene therapy products are designed for the needs of the patients who will receive them.

This means that stakeholders must be proactive to effectively incorporate patient goals and perspectives throughout the gene therapy development process. Developers of gene therapy products should survey patients all along the development path from the beginning, include patients in industry meetings, and make an active effort to create additional opportunities for patient engagement. The FDA should ensure that patients sit on advisory committees and incorporate their views into regulatory policies, including industry guidances centered on gene therapies. Finally, patient advocacy groups must devote sufficient resources to community outreach, engagement, and education on the subject of gene therapies.

This is a long-term effort that requires time and resources. But by creating a system in which ongoing, constructive conversations among communities, developers, and regulators are the standard operating model, we move closer to enacting a truly patient-focused model of gene therapy development.